Sarepta Trial Protocol Changes May Ignite Debate On Placebo Design
US FDA’s external advisors will meet May 18 to consider allowing use of central venous access lines in a study of two Duchenne muscular dystrophy drugs. Agency briefing documents show decision-making on placebo-controlled trial design was informed by problems in the clinical development of Exondys 51 (eteplirsen).
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US agency’s experts unanimously support allowing use of in-dwelling ports to aid infusions in trial of two Duchenne muscular dystrophy compounds, while also endorsing the two-year, double-blind, placebo-controlled design, which was influenced by the clinical experience with Exondys 51 (eteplirsen).
US agency’s advisory committees will consider whether ongoing trial of exon 45- and 53-skipping compounds for Duchenne muscular dystrophy should allow use of in-dwelling ports to aid infusion.
Agency reviewers believed the line between patient input and external intimidation had been crossed; early data from the Sarepta muscular dystrophy drug's flawed development program stoked patient community expectations and made regulatory review difficult.